BioMarin Pharmaceutical Inc. received a fast track designation for Phenoptin (sapropterin dihydrochloride) for phenylketonuria (PKU). Phenoptin, an investigational oral small molecule therapeutic, is currently in phase 3 clinical development.

"We are pleased that the FDA recognises that PKU is a serious disease and unmet medical need, and we look forward to working closely with the agency in our effort to develop Phenoptin as the first drug therapy for its treatment," stated Jean-Jacques Bienaime, chief executive officer of BioMarin.

"In early December of 2005, we enrolled the last patient into the on-going phase 3 clinical trial of Phenoptin, keeping us on-track to announce top-line results from the double-blind, placebo-controlled portion of this trial in late March 2006," added Bienaime.

Phenoptin is an investigational oral small molecule therapeutic for the treatment of PKU. The active ingredient in Phenoptin, sapropterin dihydrochloride, is the synthetic form of 6R-BH4, a naturally occurring enzyme cofactor. BioMarin received orphan drug designation for Phenoptin to treat PKU from both the FDA and European Medicines Agency.

PKU, a genetic disorder affecting approximately 50,000 diagnosed patients in the developed world, is caused by a deficiency of the enzyme, phenylalanine hydroxylase (PAH). PAH is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods.

BioMarin develops and commercialises innovative biopharmaceuticals for serious diseases and medical conditions.